Oncology Drug Approval - Accelerated

Bridging the gap between biomarkers and In Vitro Companion Diagnostics

MolecularMD Submits Premarketing Approval Application to the FDA for its BCR-ABL T315I Mutation Test, a Companion Diagnostic to Ponatinib, an investigational BCR-ABL inhibitor

Portland, OR, July 30, 2012 – MolecularMD Corp. today announced that it has submitted its application for Premarket Approval (PMA) of the MolecularMD BCR-ABL T315I Mutation Test to the U.S. Food and Drug Administration (FDA). This test detects the T315I mutation in BCR-ABL RNA transcripts in blood using Sanger sequencing technology. The MolecularMD test can be used to identify patients with chronic myeloid leukemia (CML) and Philadelphia positive acute lymphoblastic leukemia (Ph+ ALL) who have the T315I mutation; the test is intended as a companion diagnostic to ponatinib. Ponatinib, a BCR-ABL inhibitor developed by ARIAD Pharmaceuticals, Inc., is being submitted for approval for the treatment of patients with resistant or intolerant CML or Ph+ ALL, including those with the T315I mutation. BCR-ABL mutation testing for the PACE trial, the pivotal phase 2 trial of ponatinib, was exclusively performed by MolecularMD.

MolecularMD’s President, Dan Snyder, stated, “We have worked closely in partnership with ARIAD to perform all the mutation testing for the pivotal PACE trial of ponatinib and at the same time, complete the in vitro diagnostic test development and PMA submission. We are among the first partnerships to submit an NDA and a PMA to the FDA for simultaneous consideration, following the draft guidance set forth by the FDA in July 2011. We look forward to offering this companion diagnostic for use with ponatinib to identify CML and Ph+ ALL patients with the T315I mutation.”

ABL tyrosine kinase inhibitors (TKIs) that block deregulated BCR-ABL activity have transformed the care of CML and Ph+ ALL patients over the past decade. While second-generation ABL TKIs have demonstrated potency against a number of imatinib-resistant forms of BCR-ABL, they are ineffective against the T315I mutant. Ponatinib has potent activity against native BCR-ABL and its mutants, including T315I.

MolecularMD BCR-ABL T315I Mutation Test Details

The test service employs bidirectional Sanger sequencing to detect the T315I mutation in the predominant BCR-ABL transcripts that account for over 95% of gene fusion products in CML and Ph+ ALL cases. If approved, the Molecular MD BCR-ABL T315I Mutation Test would be the first BCR-ABL sequencing test approved by the FDA and the first service-based laboratory-developed test to be approved by the FDA as a companion diagnostic. Pending approval, the MolecularMD BCR-ABL T315I Mutation Test will facilitate identification of CML and Ph+ ALL patients who are candidates for ponatinib treatment.

About Oncology Molecular Diagnostics

Targeted cancer therapeutics account for approximately half of all cancer drug sales in the US or an estimated $25 Billion. Molecular diagnostics are used to select patients most likely to respond to a targeted therapy, as well as to monitor and manage potential resistance to therapy. Increasingly, regulators are requiring that companion diagnostic tests, which define the appropriate patient population, accompany submissions for approval of new precision cancer medicines.

About MolecularMD

MolecularMD Corporation develops and commercializes specialty molecular diagnostics for oncology applications. Its tests are designed to allow appropriate selection, monitoring and management of patients treated with molecularly-targeted cancer therapies. MolecularMD incorporates gold-standard and innovative technologies in providing its partners with the highest quality results. Assays are designed to meet clinical trial needs, and MolecularMD has appropriate systems and standards in place to enable development of companion diagnostic tests in conjunction with partners' novel anticancer agents. A private company based in Portland, Oregon, MolecularMD was founded by Brian Druker, director of the Knight Cancer Institute at Oregon Health & Science University, and Sheridan G. Snyder, entrepreneur and founder of Genzyme Corporation.